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Human growth hormone can be used successfully to treat some symptoms of cystic fibrosis, but its impact on the disease itself remains unknown, according to a new report funded by HHS' Agency for Healthcare Research and Quality (AHRQ).

Cystic fibrosis is an inherited chronic multi-organ disease, caused by a defective gene, in which the body produces thick mucus that clogs the lungs and leads to life-threatening lung infections. The disease also makes it difficult for the pancreas to work, hampering the ability to absorb food. Common signs are salty tasting skin, difficulty breathing, chronic lung infections, and poor weight gain and shorter height. The disease typically is detected in childhood, stunting the patient's growth and usually leading to early death.

The report, Effectiveness of Recombinant Human Growth Hormone (rhGH) in the Treatment of Patients with Cystic Fibrosis, finds that the use of human growth hormone increases height and weight, may improve lung functioning and may strengthen the bones of patients with cystic fibrosis. Researchers found evidence suggesting that human growth hormone therapy reduces the need for hospitalizations but could find no evidence that the therapy prolongs life or improves health-related quality of life. The use of human growth hormone was also found to raise blood sugar, which may over time lead to the development of diabetes in some patients.

"Patients with cystic fibrosis and their families have long looked for ways to manage this disease," said AHRQ Director Carolyn M. Clancy, M.D. "This report gives patients and their families excellent information that they can use, in consultation with their doctors, to make decisions about care."

The report was produced by the University of Connecticut/Hartford Hospital Evidence-based Practice Center for AHRQ, a leading federal agency conducting patient-centered outcomes research. Results of the report were published online today in the journal Pediatrics. Researchers examined published evidence from 53 unique studies of the effect of human growth hormone in cystic fibrosis patients as well as patients who did not have cystic fibrosis.

Cystic fibrosis usually is diagnosed in early childhood, and recent developments in screening can detect the disease shortly after birth. It afflicts about 30,000 children and adults in the United States. In the 1950s, few children diagnosed with cystic fibrosis lived to attend elementary school, but advances in treatment have improved the median age of survival to 37 years old.

Human growth hormone is commonly used to treat several conditions, including growth hormone deficiency, chronic kidney failure and small size in children. Human growth hormone therapy requires that patients inject the medication every day for several months.

Effectiveness of Recombinant Human Growth Hormone (rhGH) in the Treatment of Patients with Cystic Fibrosis is the newest comparative effectiveness review from AHRQ's Effective Health Care Program. The Effective Health Care Program represents a leading federal effort to compare alternative treatments for health conditions and make the findings public to help doctors, nurses, pharmacists and others work together with patients to choose the most effective treatments.

In conjunction with the new report, AHRQ will soon publish plain-language summary guides about human growth hormone in cystic fibrosis for patients, clinicians and policymakers. Summary guides on numerous clinical topics and other information and background on the Effective Health Care Program can be found here.

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